EP3458585A4 - GENE THERAPY PROCEDURES FOR AGE-RELATED ILLNESSES AND SUFFERING - Google Patents

GENE THERAPY PROCEDURES FOR AGE-RELATED ILLNESSES AND SUFFERING Download PDF

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Publication number
EP3458585A4
EP3458585A4 EP17800329.9A EP17800329A EP3458585A4 EP 3458585 A4 EP3458585 A4 EP 3458585A4 EP 17800329 A EP17800329 A EP 17800329A EP 3458585 A4 EP3458585 A4 EP 3458585A4
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EP
European Patent Office
Prior art keywords
affections
age
methods
gene therapy
related diseases
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP17800329.9A
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German (de)
English (en)
French (fr)
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EP3458585A2 (en
Inventor
Noah DAVIDSOHN
George M. Church
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Harvard University
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Harvard University
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Publication of EP3458585A2 publication Critical patent/EP3458585A2/en
Publication of EP3458585A4 publication Critical patent/EP3458585A4/en
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/50—Fibroblast growth factor [FGF]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Toxicology (AREA)
  • Cardiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Cell Biology (AREA)
  • Hospice & Palliative Care (AREA)
  • Child & Adolescent Psychology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Immunology (AREA)
EP17800329.9A 2016-05-20 2017-05-22 GENE THERAPY PROCEDURES FOR AGE-RELATED ILLNESSES AND SUFFERING Pending EP3458585A4 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662339182P 2016-05-20 2016-05-20
US201662421665P 2016-11-14 2016-11-14
PCT/US2017/033815 WO2017201527A2 (en) 2016-05-20 2017-05-22 Gene therapy methods for age-related diseases and conditions

Publications (2)

Publication Number Publication Date
EP3458585A2 EP3458585A2 (en) 2019-03-27
EP3458585A4 true EP3458585A4 (en) 2019-10-30

Family

ID=60326179

Family Applications (1)

Application Number Title Priority Date Filing Date
EP17800329.9A Pending EP3458585A4 (en) 2016-05-20 2017-05-22 GENE THERAPY PROCEDURES FOR AGE-RELATED ILLNESSES AND SUFFERING

Country Status (9)

Country Link
US (2) US12281154B2 (enExample)
EP (1) EP3458585A4 (enExample)
JP (4) JP7632835B2 (enExample)
CN (2) CN109844124B (enExample)
AU (2) AU2017268469C1 (enExample)
BR (1) BR112018073861A2 (enExample)
CA (1) CA3025020A1 (enExample)
MX (2) MX2018014256A (enExample)
WO (1) WO2017201527A2 (enExample)

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EP4653536A2 (en) * 2017-03-17 2025-11-26 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
JP7778469B2 (ja) * 2017-05-24 2025-12-02 ウニベルシダッド アウトノマ デ バルセロナ 線維芽細胞増殖因子２１（ｆｇｆ２１）コーディング配列を含むウイルス発現コンストラクト
CN119390821A (zh) 2017-08-15 2025-02-07 伊兰科美国公司 兽药用IgG Fc变体
US11530433B2 (en) * 2017-10-20 2022-12-20 President And Fellows Of Harvard College Artificial secretion peptides for heterologous protein production
US11807850B2 (en) 2017-12-06 2023-11-07 Klogenix Llc Compositions and methods for modulating gene expression
CA3110290A1 (en) * 2018-08-30 2020-03-05 Research Institute At Nationwide Children's Hospital Gene therapy for the treatment of galactosemia
BR112021006977A2 (pt) * 2018-10-18 2021-07-27 Kindred Biosciences, Inc. variantes fc com ligação alterada a receptor fc neonatal (fcrn) para uso veterinário
US12134772B2 (en) 2018-11-21 2024-11-05 Advantage Therapeutics, Inc. Increasing gene expression
CA3120923A1 (en) * 2018-11-26 2020-06-04 Universitat Autonoma De Barcelona Fibroblast growth factor 21 (fgf21) gene therapy
US11505797B2 (en) 2019-05-23 2022-11-22 Christiana Care Health Services, Inc. Gene knockout of variant NRF2 for treatment of cancer
US12203070B2 (en) 2019-05-23 2025-01-21 Christiana Care Gene Editing Institute, Inc. Gene knockout of NRF2 for treatment of cancer
JP2022536692A (ja) 2019-06-11 2022-08-18 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 調節不全の血漿カリクレインによって媒介される疾患の治療のための抗体のアデノ随伴ウイルスベクター送達
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CN115515645A (zh) * 2019-12-05 2022-12-23 哈佛学院董事及会员团体 治疗骨关节炎的方法
JP7588466B2 (ja) * 2020-01-22 2024-11-22 旭化成メディカル株式会社 パルボウイルスの生産方法
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